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Enzyme Replacement in a Human Model of Mucopolysaccharidosis IVA In Vitro and Its Biodistribution in the Cartilage of Wild Type Mice

Mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome) is a lysosomal storage disorder caused by deficiency of N-acetylgalactosamine-6-sulfatase (GALNS), an enzyme that degrades keratan sulfate (KS). Currently no therapy for MPS IVA is available. We produced recombinant human (rh)GALNS as a potenti...

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Detalles Bibliográficos
Autores principales:	Dvorak-Ewell, Melita, Wendt, Dan, Hague, Chuck, Christianson, Terri, Koppaka, Vish, Crippen, Danielle, Kakkis, Emil, Vellard, Michel
Formato:	Texto
Lenguaje:	English
Publicado:	Public Library of Science 2010
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2922370/ https://www.ncbi.nlm.nih.gov/pubmed/20808938 http://dx.doi.org/10.1371/journal.pone.0012194

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